KemPharm has changed its name to Zevra Therapeutics, a nod to the rare disease community. 

The new name, Greek for “zebra,” is the internationally recognized symbol for the community and signifies an intensified focus on developing groundbreaking therapies for rare diseases with limited or no available treatments, according to a press release from the company. 

“The adoption of the name Zevra, the Greek word for zebra, is an important step in our journey to become a leading rare disease company since the zebra is the recognized symbol of the rare disease community around the world,” says Richard W. Pascoe, CEO of Zevra, in a press release. “With the launch of our new company name in anticipation of Rare Disease Day next week, we renew our unwavering commitment to stand with this important community as we pursue our primary mission to deliver life-changing treatments to people with rare conditions, their families, and caregivers who desperately need better options.”

Zevra is currently advancing KP1077, a product candidate based on Zevra’s prodrug of d-methylphenidate, serdexmethylphenidate, which is being evaluated in a phase 2 trial for the treatment of idiopathic hypersomnia, a rare sleep disorder affecting fewer than 200,000 people in the US, according to the National Institutes of Health. 

Pending the results from that trial, the company plans to conduct a pivotal phase 3 study in idiopathic hypersomnia, with the potential to study an expanded indication in narcolepsy.

Additionally, in May 2022, Zevra, then KemPharm, acquired arimoclomol, an orally-delivered, investigational product candidate for treating Niemann-Pick type C disease, from Orphazyme A/S as part of the company’s long-term vision to evolve into a commercially driven rare disease therapeutics company. Niemann-Pick type C is a rare disease with no currently approved treatments in the US, which primarily affects children and is often fatal.

“The Zevra team is actively leveraging their deep scientific and clinical expertise and strategic approach to overcome drug development and regulatory barriers to advance much-needed solutions to the people who need them,” says Matthew R. Plooster, chairman of the board of directors, in a press release. “Moreover, we have the leadership team and financial resources to get our promising rare disease drug candidates over the regulatory approval finish line and into the US market, which the board firmly believes will be a key value-driver for patients and shareholders alike.”

The company’s common shares will trade on the Nasdaq Global Select Market under the new ticker symbol “ZVRA” starting on or about March 1, 2023.

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